Thursday, December 13, 2012
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Yeast cell lines for
producing improved enzyme replacement therapies to treat Pompe's disease
strains of the yeasts Yarrowia lipolytica and Pichia pastoris
may be useful for producing Pompe's disease enzyme replacement therapies that
have better tissue uptake than unmodified acid a-glucosidase (GAA). The yeast strains
were modified to express a highly glycosylated form of human recombinant GAA,
the lysosomal enzyme that is absent or dysfunctional in Pompe's disease. GAA
was then purified from the yeast and treated with two bacterial glycosidase
enzymes to generate GAA highly enriched in mannose-6-phosphate (M-6-P), a
carbohydrate that targets GAA to the lysosome. In vitro, the
M-6-P-enriched GAA was taken up by Pompe's disease patient fibroblasts in
larger quantities than unmodified GAA. In a mouse model of Pompe's disease,
the M-6-P-enriched GAA cleared heart and thigh muscle of glycogen better than
unmodified GAA. Next steps carried out by Oxyrane
U.K. Ltd. include animal toxicology studies.
Myozyme and Lumizyme are human
recombinant GAA enzyme replacement therapies marketed by Sanofi's
Corp. unit to treat Pompe's disease (see Muscling up on
Myozyme, page 5).
Published online Dec. 13, 2012
licensing status undisclosed
P. et al. Nat. Biotechnol.; published online Nov. 18, 2012;
Contact: Wouter Vervecken, Oxyrane U.K. Ltd., Manchester,
Contact: Nico Callewaert, Flanders
Institute for Biotechnology (VIB), Ghent, Belgium
Contact: Han Remaut, same affiliation as above
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